Early Wins: RegenXBio's Gene Therapy Helps Duchenne Patients Walk Stronger, Longer

RegenXBio Inc. RGNX revealed new interim data on Thursday from the Phase 1/2 AFFINITY DUCHENNE trial.

Updates include positive functional, safety, and biomarker data for RGX-202, REGENXBIO’s investigational gene therapy for Duchenne muscular dystrophy.

The data update looked at five patients who were six to 12 years old when they received the gene therapy RGX-202.

The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202.

Also Read: Regenxbio Touts Positive Biomarker Data From Duchenne Muscular Dystrophy Gene Therapy Trial

RGX-202 continues to demonstrate evidence of positively impacting disease trajectory, with dose level 2 participants demonstrating improved performance on tests of time to stand, 10-meter walk-run, and time to climb in the five patients with nine months of follow-up and the four patients with 12 months of follow up.

At 9 months, RGX-202 participants demonstrated improved function and exceeded external controls on all measures. On the North Star Ambulatory Assessment (NSAA, functional motor abilities scale), RGX-202 recipients improved an average of 4 points from baseline and 4.8 points compared to natural history.

Four out of the five participants reached 12 months post-dosing. Results at 12 months are similar to those seen at 9 months.

RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months.

All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. On NSAA, RGX-202 recipients improved an average of 4.5 points from baseline and 6.8 points compared to natural history.

RegenXBio expects to share topline data for RGX-202 in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.

Commercial readiness activities are underway to support an expected launch in 2027.

In March, Sarepta Therapeutics, Inc. SRPT shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.

The company reported a patient death following treatment with Elevidys, having suffered acute liver failure.

Sarepta and Roche announced a therapeutic clinical hold on Study 104, Study 302, and Study 303 in the EU until the analysis of the cause of death is complete.

Price Action: RGNX stock is down 11.2% at $8.95 at the last check on Thursday.

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